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Cat. No.	Product Name	Target	Signaling Pathways
T39516	Bocidelpar	PPAR	DNA Damage/DNA Repair; Metabolism
	Bocidelpar 是一种有效的过氧化物酶体增殖物激活受体 delta 调节剂，可改善杜兴肌营养不良肌肉细胞中的线粒体生物合成和功能。
T7837	SR8278	Others	Others
	SR8278 是竞争性核血红素受体REV-ERB 合成拮抗剂，能够抑制 REV-ERBα 转录活性(EC50:0.47 μM)。它可用于调节生物体内的代谢和研究生物节律。
T17217	Vamorolone 地塞米松EP杂质E,VBP15	Glucocorticoid Receptor; NF-κB	Endocrinology/Hormones; NF-κB
	Vamorolone (VBP15) 是一种首创的，具有口服活性的解离性类固醇 (dissociative steroidal) 抗炎药和膜稳定剂。它抑制 (NF-κB) 抑制作用，并降低了激素的影响。它改善肌营养不良，无副作用。
T3424	Ezutromid BMN 195,VOX-C1100,依珠曲米,SMT C1100	Others	Others
	Ezutromid (BMN 195) 是一种具有口服活性的、首创的苯并恶唑 utrophin 调节剂，EC50=0.91 μM。它抑制人肝微粒体 CYP1A2 酶活性，IC50=5.4 μM。它可用于 Duchenne 型肌营养不良症 (DMD) 的研究。
T15198	Edasalonexent CAT-1004	NF-κB	NF-κB
	Edasalonexent (CAT-1004) 是一种可口服的 NF-κB 抑制剂，可用于改善杜氏肌营养不良症。
T9821	ART-CHEM-BB B025267 2-(4-二乙基氨基苯基)-6-甲基-5-苯并三唑胺	Others	Others
	ART-CHEM-BB B025267 是 utrophin 生成的上调剂，EC50 为 1.8 μM，可用于治疗 Duchenne 肌营养不良症的研究。
T1805	Ataluren PTC124	CFTR; Autophagy	Autophagy; Membrane transporter/Ion channel
	Ataluren (PTC124) 是一种可口服的 CFTR-G542X 无义等位基因抑制剂。
T38554	Eteplirsen 依特立生；依特普森,AVI 4658
	Eteplirsen (AVI 4658) is a synthetic antisense oligonucleotide utilized in research for Duchenne muscular dystrophy.
T34425	RTC13 Read-through compound 13,RTC 13,RTC-13
	RTC13, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.
T39517	Suvodirsen WVE-210201
	Suvodirsen (WVE-210201) is an oligonucleotide compound with potential applications in the study of Duchenne muscular dystrophy (DMD).
T34426	RTC14 Read-through compound-14,RTC 14,RTC-14,Read-through compound 14
	RTC14, as a premature termination codon (PTC) readthrough inducer, can act by restoring dystrophin expression and improving muscle function in the mdx mouse model for Duchenne muscular dystrophy.
T38860	Golodirsen Golodirsen,SRP-4053
	Golodirsen (SRP-4053) is a phosphorodiamidate morpholino oligomer (PMO) designed to selectively bind and target exon 53 of dystrophin pre-mRNA, with applications in the investigation of Duchenne muscular dystrophy (DMD).
T60594	OX01914
	OX01914 是一种水溶性和细胞膜可渗透的 utrophin 调节剂，能够上调 utrophin 蛋白水平，EC50值为 20.5 μM。 OX01914 可用于研究杜氏肌肉营养不良症(DMD)。
T66481	TG-693
	TG693, an orally available inhibitor of CLK1, promotes the skipping of the endogenous mutated exon 31 in Duchenne muscular dystrophy (DMD) patient-derived cells and increased the production of the functional exon 31-skipped dystrophin protein.
T39455	Viltolarsen NS-065,NCNP-01
	Viltolarsen (NS-065/NCNP-01) is a phosphorodiamidate morpholino antisense oligonucleotide that specifically targets the splicing of exon 53 in the dystrophin gene. It is utilized in research related to Duchenne muscular dystrophy (DMD).
T61645	Utrophin modulator 1
	Utrophin Modulator 1 (UM1) is a highly effective agent that upregulates utrophin protein levels, displaying an EC 50 of 0.11 μM. Its application in the investigation of Duchenne Muscular Dystrophy (DMD) has been well-established [1].
T14129	Adenylosuccinic acid Aspartyl adenylate,Adenylosuccinate	Others	Others
	Adenylosuccinic acid (Adenylosuccinate; Aspartyl adenylate) is a purine ribonucleoside monophosphate and plays a role in nucleotide cycle metabolite. Adenylosuccinic acid has the potential for the study of duchenne muscular dystrophy(DMD)[1] and can be co
T33595	Naproxcinod AZD3582,Nitronaproxen,AZD-3582,Naproxen-N-butyl nitrate,HCT3012,AZD 3582
	Naproxcinod, a nitric oxide-releasing NSAID derivative, significantly improved skeletal muscle strength and fatigue resistance in sedentary and exercise mice, reducing inflammatory infiltration and fibrotic deposition in the myocardium and diaphragm. In a
T38859	Casimersen SRP-4045
	Casimersen (SRP-4045) is an antisense oligonucleotide, specifically belonging to the phosphorodiamidate morpholino oligomer subclass. It binds to exon 45 of dystrophin pre-mRNA, effectively restoring the open-reading frame by skipping exon 45. This action ultimately leads to the production of a functional dystrophin protein, albeit internally truncated. Casimersen finds utility in Duchenne muscular dystrophy (DMD) research.
T37449	SR 12460
	SR 12460 is a mimetic of the IκB kinase β (IKKβ) NEMO/IKKγ-binding domain (NBD) that inhibits the protein-protein interaction between the IKK complex subunits NF-κB essential modulator (NEMO/IKKγ) and IKKβ.1It inhibits TNF-α-induced NF-κB activation in a reporter assay when used at concentrations ranging from 25 to 150 μM. It reduces LPS-induced production of IL-6 in RAW 264.7 cells. SR 12460 (30 mg/kg) improves grip strength in themdxmouse model of Duchenne muscular dystrophy. 1.Zhao, J., Zhang...
T73919	Adenylosuccinic acid tetraammonium
	Adenylosuccinic acid tetraammonium（Adenylosuccinate; Aspartyl adenylate）是一种在核苷酸循环代谢中起作用的口服活性嘌呤核糖核苷单磷酸。该化合物可通过腺苷琥珀酸裂解酶转化为富马酸，展现出在杜氏肌营养不良症（DMD）研究中的应用潜力。
T75137	BAY-747
	BAY-747 (BAY 1165747) 为一种通过口服生效且具有脑渗透性的sGC刺激剂。它能可逆地逆转L-NAME所诱导的记忆障碍并在大鼠中增强认知功能。BAY-747还可在有意识状态下降低正常血压及自发性高血压大鼠(SHR)的血压。此外，BAY-747在mdx/mTRG2小鼠模型中改善了与Duchenne肌营养不良症(DMD)相关的骨骼肌功能。

化合物

Cat.No: T39516

Target: PPAR

Cat.No: T7837

Target: Others

Cat.No: T17217

Synonym: 地塞米松EP杂质E,VBP15

Target: Glucocorticoid Receptor, NF-κB

Cat.No: T3424

Synonym: BMN 195,VOX-C1100,依珠曲米,SMT C1100

Target: Others

Cat.No: T15198

Synonym: CAT-1004

Target: NF-κB

ART-CHEM-BB B025267

Cat.No: T9821

Synonym: 2-(4-二乙基氨基苯基)-6-甲基-5-苯并三唑胺

Target: Others

Cat.No: T1805

Synonym: PTC124

Target: CFTR, Autophagy

Cat.No: T38554

Synonym: 依特立生；依特普森,AVI 4658

Cat.No: T34425

Synonym: Read-through compound 13,RTC 13,RTC-13

Cat.No: T39517

Synonym: WVE-210201

Cat.No: T34426

Synonym: Read-through compound-14,RTC 14,RTC-14,Read-through compound 14

Cat.No: T38860

Synonym: Golodirsen,SRP-4053

Cat.No: T60594

Cat.No: T66481

Cat.No: T39455

Synonym: NS-065,NCNP-01

Utrophin modulator 1

Cat.No: T61645

Adenylosuccinic acid

Cat.No: T14129

Synonym: Aspartyl adenylate,Adenylosuccinate

Target: Others

Cat.No: T33595

Synonym: AZD3582,Nitronaproxen,AZD-3582,Naproxen-N-butyl nitrate,HCT3012,AZD 3582

Cat.No: T38859

Synonym: SRP-4045

Cat.No: T37449

Adenylosuccinic acid tetraammonium

Cat.No: T73919

Cat.No: T75137

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